Roche has announced new four-year data from the RHONE-X extension study, demonstrating Vabysmo’s potential as an effective long-term treatment for diabetic macular edema (DME). This study, presented at the American Society of Retina Specialists (ASRS) 2024 Annual Meeting in Stockholm, Sweden, met all primary endpoints, showing that Vabysmo was well tolerated and effective in people with DME who received treatment for up to four years. Vabysmo, a first-in-class, high-sustained factor VIII replacement therapy, offers a unique therapeutic approach by providing continuous delivery of medicine through a refillable implant. These new results reinforce Vabysmo’s potential to become a standard of care treatment for DME, a prevalent condition affecting millions worldwide.
The RHONE-X study is the largest long-term extension dataset in DME to date, showcasing Vabysmo’s sustained efficacy and safety profile. More than 90% of patients had an absence of DME after four years, a significant achievement for those living with this condition. Nearly 80% of participants extended their treatment intervals to every three or four months, demonstrating the drug’s ability to reduce the treatment burden while maintaining vision improvements and retinal dryness. These results build on the primary one-year analyses of the Pagoda and Pavilion studies, which also showed Vabysmo’s effectiveness in treating DME and diabetic retinopathy (DR).
Primary and Exploratory Endpoint Achievements
In the RHONE-X study, Vabysmo maintained vision gains and anatomical improvements over the four-year period. The primary endpoint showed no inhibitor development to factor VIII, while the exploratory analysis revealed that 95% of patients did not need additional treatment with supplemental injections. The central subfield thickness (CST), a measure of retinal swelling, remained stable, indicating sustained retinal drying. This consistency in outcomes highlights Vabysmo’s potential to provide long-term benefits to patients with DME.
The US Food and Drug Administration (FDA) has accepted Roche’s supplemental Biologics License Application (sBLA) for Vabysmo for the treatment of DME and DR, based on the one-year results from the Pagoda and Pavilion studies. Vabysmo is already approved in nearly 100 countries for DME and neovascular or ‘wet’ age-related macular degeneration (nAMD). It is also approved in several countries, including the United States and Japan, for the treatment of macular edema following retinal vein occlusion. More than four million doses of Vabysmo have been distributed worldwide since its initial US approval in 2022.
Roche’s Commitment to Ophthalmology
Roche is committed to pioneering therapies that save people’s eyesight from the leading causes of vision loss. The company’s extensive retina pipeline is led by scientific innovation and insights from patients with eye diseases. Roche’s pipeline includes treatments for multiple vision-threatening conditions, such as diabetic eye diseases, geographic atrophy, and autoimmune conditions. The company has brought breakthrough ophthalmic treatments to market, including Susvimo, a refillable eye implant for nAMD, and Vabysmo, the first bispecific antibody approved for the eye.
Roche continues to expand its clinical development program for Vabysmo with several Phase III and Phase IV studies. These include AVONELLE-X, an extension study of TENAYA and LUCERNE, evaluating the long-term safety and tolerability of Vabysmo in nAMD. Other studies like ELEVATUM and SALWEEN aim to understand Vabysmo’s efficacy in underrepresented populations and specific subpopulations. Additionally, the VOYAGER study, a global real-world data collection platform, supports further understanding of retinal conditions with high unmet needs.
About Diabetic Macular Edema (DME)
Diabetic macular edema (DME) is a vision-threatening retinal condition affecting approximately 29 million people globally. DME occurs when damaged blood vessels leak into and cause swelling in the macula, the central area of the retina responsible for sharp vision. This condition is associated with blindness and decreased quality of life if left untreated. The prevalence of DME is expected to grow as diabetes becomes more common worldwide.
Vabysmo is the first bispecific antibody approved for the eye, targeting and inhibiting two signaling pathways linked to vision-threatening retinal conditions by neutralizing angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A). These pathways contribute to vision loss by destabilizing blood vessels and increasing inflammation. Vabysmo is designed to stabilize blood vessels and reduce leakage. It is approved for use in nAMD and DME in nearly 100 countries and for treating macular edema following retinal vein occlusion in several countries.
Founded in 1896, Roche has grown into the world’s largest biotechnology company and a leader in in-vitro diagnostics. Roche pursues scientific excellence to discover and develop medicines and diagnostics that improve and save lives. The company is committed to personalized healthcare and transforming healthcare delivery. Roche has been recognized as one of the most sustainable companies in the pharmaceuticals industry by the Dow Jones Sustainability Indices for fifteen consecutive years. The company combines strengths in Diagnostics and Pharma with data insights from clinical practice to provide the best care for each person.
Resource: Roche, July 17, 2024
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